CORDIS Project
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This project focuses on developing a pipeline called EnhancerDesign to create specific human enhancers for gene therapy. By optimizing the design and testing of these enhancers, the project aims to improve the safety and effectiveness of gene therapy treatments for various diseases.
Gene therapy promises to become a durable clinical option for a whole range of diseases, including monogenic hereditary diseases, cancer and neurodegenerative disorders.
However, the development of new gene therapy-based treatments strongly depends on regulatory drivers that provide cell-type specific expression of the transgene, at high enough levels, yet without causing genotoxicity.The aim of the ERC PoC proposal is to validate a state-of-the-art pipeline, called EnhancerDesign, to design sma…
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